SRX246: Safety, Tolerability, and Activity in Irritable subjects with HD

Huntington's disease (HD) is an inherited disease that results from expansion of a trinucleotide (CAG, cytosine/adenine/guanine) repeat that encodes a polyglutamine tract in the huntingtin gene and gradually damages neurons.  Overtime this cellular loss affects voluntary motor control, results in involuntary movements (chorea), and causes cognitive decline.  Psychiatric symptoms, including irritability, are commonly seen in HD, and are quite distressing for patients and their family. Preclinical pharmacology studies suggest that SRX246, a first in-class vasopressin 1a (V1a) receptor antagonist, has potential as a novel therapeutic agent for major neuropsychiatric symptoms seen in HD patients.

This research study is being conducted to test the tolerability and further examine the safety of SRX246 in irritable subjects with early symptomatic HD when it is given orally at doses up to 160 mg twice daily compared to placebo. Approximately 150 HD patients will be screened to enroll 108 subjects at NeuroNEXT sites across the United States.   

Azevan Pharmaceuticals, Inc. is the Sponsor organization for this clinical trial and is conducting the trial through the NeuroNEXT Network under a Cooperative Agreement with NINDS (U44 NS090616).

Protocol Principal Investigator: 
Contact Information: 

CCC Project Managers: Catherine Gladden, Andrea Ariza, Brenda Thornell

DCC Project Coordinators: Michele Costigan, Holly Ernst, BA, CCRC

  • Michael J. Brownstein, Neal G. Simon, Jeffrey D. Long, Jon Yankey, Hilda T. Maibach, Merit Cudkowicz, Christopher Coffey, Robin A. Conwit, Codrin Lungu, Karen E. Anderson, Steven M. Hersch, Dixie J. Ecklund, Eve M. Damiano, Debra E. Itzkowitz, Shifang Lu, Marianne K. Chase, Jeremy M. Shefner, Andrew McGarry, Brenda Thornell, Catherine Gladden, Michele Costigan, Padraig O’Suilleabhain, Frederick J. Marshall, Amy M. Chesire, Paul Deritis, Jamie L. Adams, Peter Hedera, Kelly Lowen, H. Diana Rosas, Amie L. Hiller, Joseph Quinn, Kellie Keith, Andrew P. Duker, Christina Gruenwald, Angela Molloy, Cara Jacob, Stewart Factor, Elaine Sperin, Danny Bega, Zsazsa R. Brown, Lauren C. Seeberger, Victor W. Sung, Melanie Benge, Sandra K. Kostyk, Allison M. Daley, Susan Perlman, Valerie Suski, Patricia Conlon, Matthew J. Barrett, Stephanie Lowenhaupt, Mark Quigg, Joel S. Perlmutter, Brenton A. Wright, Elaine Most, Guy J. Schwartz, Jessica Lamb, Rosalind S. Chuang, Carlos Singer, Karen Marder, Joyce A. Moran, John R. Singleton, Meghan Zorn, Paola V. Wall, Richard M. Dubinsky, Carolyn Gray and Carolyn Drazinic Safety and Tolerability of SRX246, a Vasopressin 1a Antagonist, in Irritable Huntington’s Disease Patients—A Randomized Phase 2 Clinical Trial. Journal of Clinical Medicine20209(3682)1-14.
    PubMed: PMC7696926